News Release

Scripps Research Scientist Receives $98,000 Grant from Factor Foundation to Study Hemophilia A

Factor Also to Sponsor Vintage Mustang Car Show June 11

La Jolla, CA, May 5, 2005—Assistant Professor Andrew Gale, Ph.D., an investigator in the Department of Molecular and Experimental Medicine at The Scripps Research Institute, has received a grant of $98,769 from the Factor Foundation of America to study hemophilia A, a bleeding disease that strikes some 17,000 Americans.

The Factor Foundation, based in Boca Raton, is a national nonprofit organization dedicated to facilitating grants for research, education, and clinical support for hemophilia and other bleeding disorders.

In addition to its grant support, the foundation is hosting its first public fundraising event, "Dave's Memory Revs On," at the Moroso Motorsports Park in Palm Beach Gardens, Florida, on June 11. All funds from the show of vintage Ford Mustang cars, commemorating the foundation's founder, Dave Madeiros, will benefit Scripps Florida, the Institute's new operation in Palm Beach County.

"The Scripps Research Institute shares the Factor Foundation of America's concern for the thousands of Americans who suffer from debilitating, sometimes fatal, bleeding disorders," said Institute President Richard A. Lerner. "We deeply appreciate the foundation's generous support of our work on hemophilia A, both through the research grant and what is sure to be a festive day for families in Palm Beach County, our new neighbors."

The Research on Hemophilia A

Gale and his colleagues have already made an important discovery that they hope will lead to an improved therapy for hemophilia A. They engineered a stable form of the protein Factor VIII, which is a coagulatory blood protein that hemophiliacs have little or no ability to produce themselves.

Hemophilia A is often treated with infusions of pre-activated Factor VIII, and several forms of the treatment are already on the market. These infusions provide patients with the normal inactive form of the protein, and when that person gets cut, other proteins in the body can then process the inactive Factor VIII into its active form, called "Factor VIIIa".

However, once activated, Factor VIIIa is highly unstable and tends to fall apart in the bloodstream. This instability is an important feature of normal coagulation because it prevents the clotting process from getting out of control. In hemophiliacs being treated with Factor VIIIa, however, its instability is a problem because, as its pieces dissociate, the protein infusion loses its potency.

Gale and his colleagues made a more stable form of Factor VIIIa by engineering the protein so that its individual pieces are linked together with what are known as "disulfide bonds." These prevent the subunits from falling apart—sort of like handcuffing them together on the molecular level.

"Our Factor VIIIa is very stable," said Gale, adding that as a therapy, this increased stability could improve the effectiveness of Factor VIII infusions by reducing the number and amount of such infusions a hemophiliac would need.

But Gale and his colleagues' initial discovery a few years ago left open the question of the effect of the new stabilized form of Factor VIIIa on the body. The scientists have looked at its stability in the test tube, but further studies are needed to determine whether the body could handle the new form of the protein.

Now, the Factor Foundation grant will pay for some of the preliminary pre-clinical experiments aimed at testing how effective the new form of Factor VIIIa is in laboratory rodents. If data from these experiments look promising, then the next step would be to consider human trials.

"This is crucial support that is arriving at the best possible moment in this research," said Gale. "We want to see how well my engineered, stabilized Factor VIII works, and the Factor Foundation is making that possible."

"Dave's Memory Revs On"

The vintage Mustang show will be held from 10 a.m. to 4 p.m., Saturday, June 11 at the Moroso Motorsports Park in Palm Beach Gardens. According to Factor Foundation executive director Kim Madeiros, the event will provide South Florida families, Mustang collectors and admirers, and friends of Scripps Florida the opportunity to learn more about the biomedical research institution while having fun and raising money for hemophilia research.

"Thirteen dollars of every $15 adult admission tickets sold will be given by Moroso Motorsports Park to the Factor Foundation to support Scripps research," said Madeiros. "And additional funds from a raffle of a 2005 Ford Mustang will increase the amount raised to what I hope will exceed $100,000."

Private and corporate sponsors will cover the costs of the event.

Jeffrey W. Kelly, Ph.D., vice president for academic affairs and dean of the Kellogg School of Science and Technology at the Scripps Research campus in La Jolla, California, and an owner and racer of classic cars, will participate as a driver and guest of honor at the event. He will be joined by scientists, staff members and their families from Scripps Florida's temporary facilities on the Jupiter campus of Florida Atlantic University.

The Scripps Research Institute in La Jolla, California, and Palm Beach County, Florida, is one of the world's largest, independent, non-profit biomedical research organizations. It stands at the forefront of basic biomedical science that seeks to comprehend the most fundamental processes of life. Scripps Research is internationally recognized for its research into immunology, molecular and cellular biology, chemistry, neurosciences, autoimmune, cardiovascular, and infectious diseases, and synthetic vaccine development.

The Scripps Research Institute employs approximately 3,000 scientists, postdoctoral fellows, scientific and other technicians, doctoral degree graduate students, and administrative and technical support personnel in 14 buildings overlooking the Pacific Ocean in La Jolla, a part of the City of San Diego.

Scripps Florida will be a 350,000 square-foot, state-of-the-art biomedical research facility to be built on 100 acres of undeveloped land in Palm Beach County. Scripps Florida will focus on basic biomedical science, drug discovery, and technology development, employing more than 500 researchers and support staff by 2010. Palm Beach County and the State of Florida have provided start-up economic incentives for development, building, staffing, and equipping the campus.

Scripps Florida is now operating with more than 100 researchers and technicians at a 40,000 square-foot facility on the campus of Florida Atlantic University in Jupiter.

For more information contact:Office of Communications10550 North Torrey Pines RoadLa Jolla, California 92037press@scripps.edu